With the important announcement on October 12, 2017 of FDA approval for a Phase 3 clinical trial for trofinetide, Rettsyndrome.org has shown that we can fix the biology of Rett syndrome. Together, we have moved one step closer to bringing prescribable treatments and a cure to the thousands impacted by Rett syndrome. This would not have been possible without the generous support of our donors and active participation of families. Together, we ARE making a difference.

Because of Rettsyndrome.org’s proprietary Scout Program, we are able to identify drugs with the highest potential benefit to those suffering from Rett syndrome at an unprecedented speed. Because of Rettsyndrome.org’s funding, there are now three compounds in clinical trials — more than at any other time in the history of Rett syndrome!The ultimate cure will require two things: fixing biology AND resetting neurology. In addition to developing safe and effective compounds, Rettsyndrome.org is committed to accelerating research in neuro-habilitation to make up for lost milestones and to set development back on track once the biology is repaired. The complexity of Rett syndrome requires work in both these areas.

We invite you to partner with us as we continue to make progress and deliver groundbreaking research results. Your financial support is crucial to our continued success. Together, we will deliver our ultimate goal of a cure for Rett syndrome.